In a strategic move beyond liver-focused therapies, Intellia Therapeutics and Regeneron Pharmaceuticals have announced the expansion of their CRISPR collaboration. Following their 2016 partnership, the two companies achieved a milestone in 2021 with first-in-human gene editing results.

This expanded collaboration aims to develop in vivo CRISPR-based therapies for neurological and muscular diseases. Regeneron will contribute its proprietary adeno-associated virus vectors, while Intellia will bring its Nme2 CRISPR/Cas9 systems adapted for viral vector delivery.

Unlike traditional partnerships, no upfront payments are involved. Instead, the companies will focus on two in vivo non-liver targets initially. Intellia will lead the editing methodology, and Regeneron will handle targeted viral vector delivery.

Each company will independently lead development and commercialization for one target, with an option for the other to join in a 50/50 co-development and co-commercialization agreement. The collaboration, which began in 2016, has undergone revisions, with the latest extension until April 2024.

Their groundbreaking achievement so far includes the in vivo CRISPR/Cas9 genome editing candidate NTLA-2001 for transthyretin amyloidosis, demonstrating a reduction of serum levels of transthyretin, a key biomarkers for the disease. As Intellia and Regeneron pioneer CRISPR-based therapies beyond the liver, the scientific community anticipates potential breakthroughs in treating neurological and muscular diseases.

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